Major US report supports human genome editing – BioNews

An influential advisory group has given cautious support to the idea of making heritable changes to the human genome in order to treat or prevent disease.

The report, published by the US-based National Academy of Sciences (NAS) and the National Academy of Medicine (NAM), concluded that using genome-editing technology, such as CRISPR/Cas9, to make alterations to the germline would be acceptable if the intention was to treat or prevent serious genetic disease or disorders, and the procedure was proven to be safe.

The authors also provided a list of criteria which should be met before any specific changes are made, including the absence of reasonable alternatives and restricting editing to genes known to cause or strongly predispose people to serious disease.

The ethical implications of altering the human germline has been the subject of intense discussion in recent years, with calls for such work to be put on hold until the process of genome editing is better understood. Indeed, just a month before the publication of the NAS/NAM report, the American College of Medical Genetics and Genomics urged caution and stated their opinion that 'genome editing in the human embryo is premature' (see BioNews 887).

The NAS and NAM also participated in a genome-editing summit held in Washington in 2015, which concluded that it would be 'irresponsible' to currently proceed with germline editing (see BioNews 831).

The latest NAS/NAM report represents a more permissive view of using genome editing to alter the human genome, and has been broadly welcomed by researchers. Dr Sarah Chan, from the Usher Institute for Population Health Sciences and Informaticsat theUniversity of Edinburgh, said: 'It is encouraging that the NAS/NAM report considers heritable human genome editing as "a realistic possibility" rather than ruling it out altogether.

'Genome-editing technology holds tremendous potential benefits but the concerns around so-called "germline genome editing" must be taken into consideration; the report, in acknowledging the prospects for developing this technology, paves the way for these discussions to continue,' she added.

However, there are concerns that allowing the human genome to be edited to prevent disease could be the top of a slippery slope, ending with the genome being altered for other, non-medical reasons, particularly as potential therapies for some genetic conditions could also be used to 'enhance' people without the condition. For example, a genetic change developed to treat muscular dystrophy might also be used to make a healthy person more muscular.

Robert Meadowcroft, chief executive of Muscular Dystrophy UK, addressed these concerns, saying: 'We understand that some people may have a concern that this technique could lead to "designer babies", but we have confidence in the UK's strong regulatory and ethical system to deliver the necessary framework to safeguard against this.'

The NAS/NAM report recommended not proceeding with genome editing for purposes other than the prevention or treatment of disease, and called for public debate on the use of somatic genome editing for non-medical purposes.

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Major US report supports human genome editing - BioNews